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    Summary
    EudraCT Number:2015-000389-69
    Sponsor's Protocol Code Number:UX007G-CL202
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2017-03-27
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2015-000389-69
    A.3Full title of the trial
    An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome
    Estudio de extensión abierto para evaluar la seguridad y eficacia a largo plazo de UX007 en sujetos con síndrome de deficiencia del transportador de glucosa de tipo 1 (SD Glut1)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A trial to assess the long term safety and efficacy of UX007 in subjects who have already participated in a UX007 study.
    Un ensayo para evaluar a largo plazo la seguridad y eficacia de UX007 en pacientes que ya han participado en un estudio con UX007
    A.4.1Sponsor's protocol code numberUX007G-CL202
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02599961
    A.5.4Other Identifiers
    Name:EMA/190573Number:Unique Product Identifier (UPI)
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorUltragenyx Pharmaceutical Inc
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportUltragenyx Pharmaceutical Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUltragenyx Pharmaceutical Inc
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address60 Leveroni Court
    B.5.3.2Town/ cityNovato
    B.5.3.3Post codeCA 94949
    B.5.3.4CountryUnited States
    B.5.4Telephone number+3493 600 97 33
    B.5.6E-mailux007G-CL202@ultragenyx.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU /3/15/1495
    D.3 Description of the IMP
    D.3.1Product nameTriheptanoin
    D.3.2Product code UX007
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNno disponible
    D.3.9.1CAS number 620-67-7
    D.3.9.2Current sponsor codeUX007
    D.3.9.3Other descriptive nameTRIHEPTANOINA
    D.3.9.4EV Substance CodeSUB129584
    D.3.10 Strength
    D.3.10.1Concentration unit g/ml gram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number96
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Glucose Transporter Type 1 deficiency syndrome
    síndrome por deficiencia del transportador de glucosa de tipo 1
    E.1.1.1Medical condition in easily understood language
    Glucose transporter type 1 (Glut1) deficiency syndrome is a rare genetic metabolic disorder characterized by deficiency of a protein that is required for glucose to cross the blood-brain barrier
    El síndrome por deficiencia del transportador de glucosa de tipo 1 (Glut1) es una enfermedad metabólica rara caracterizada por la falta de una proteina necesaria para que la glucosa llegue al cerebro
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.1
    E.1.2Level LLT
    E.1.2Classification code 10061032
    E.1.2Term Carbohydrate transport disorder
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluate the long-term safety of UX007 in Glut1 DS subjects
    Evaluar la seguridad a largo plazo de UX007 en sujetos con SD Glut1
    E.2.2Secondary objectives of the trial
    Evaluate the long-term effect of UX007 efficacy on seizures associated with Glut1 DS
    Evaluate the long-term effect of UX007 on health related quality of life related to Glut1 DS
    Evaluar la el efecto a largo plazo de la eficacia de UX007 sobre las convulsiones asociadas al SD Glut1
    Evaluar el efecto a largo plazo de UX007 sobre la calidad de vida relacionada con la salud asociada al SD Glut1
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1)Diagnosis of Glut1 DS as indicated by cerebrospinal fluid glucose concentration, erythrocyte 3-O-methyl-D-glucose uptake assay, or molecular genetic testing obtained from medical records.
    2)Males and females, aged at least 1 year at the time of informed consent
    3)Completion of UX007G-CL201 study (NCT01993186). Glut1 DS patients who received UX007/triheptanoin treatment as part of other clinical studies, ISTs, or expanded access/compassionate use treatment programs may be eligible at the discretion of the sponsor.
    4)Provide written informed consent or verbal assent (if possible) with written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures
    5)Must, in the opinion of the investigator, be willing and able to complete all aspects of the study, and comply with accurate completion of the seizure diary
    6)Females of childbearing potential must have a negative urine pregnancy test at Baseline and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have not experienced menarche, are post-menopausal (defined as having no menses for at least 12 months without an alternative medical cause), or are permanently sterile due to total hysterectomy, bilateral salpingectomy, or bilateral oophorectomy.
    7)Participants of child-bearing potential or fertile males with partners of child-bearing potential who are sexually active must consent to use a highly-effective method of contraception as determined by the investigator from the period following the signing of the informed consent through 30 days after last dose of study drug.
    1) Diagnóstico de SD Glut1 según la concentración de glucosa en el líquido cefalorraquídeo,el ensayo de captación de 3-O-metil-D-glucosa en eritrocitos o pruebas genéticas moleculares recogidas de la historia clínica.

    2) Hombres y mujeres, de 1 año de edad como mínimo en el momento de la obtención del consentimiento informado.

    3) Haber completado el estudio UX007G-CL201 (NCT01993186). Los pacientes con SD Glut1 que hayan recibido tratamiento con UX007/triheptanoína como parte de otros estudios clínicos, EPI, o programas de acceso ampliado/uso compasivo del tratamiento podrán participar en el estudio si el promotor lo estima oportuno.

    4) Proporcionar el consentimiento informado por escrito o bien un asentimiento verbal (si es posible) con un consentimiento informado por escrito otorgado por un representante legalmente autorizado, después de haber explicado la naturaleza del estudio y antes de realizar cualquier procedimiento previo relacionado con la investigación.

    5) Sujeto que, en opinión del investigador, esté dispuesto y sea capaz de realizar todos los aspectos del estudio y cumplimentar con exactitud el diario de convulsiones.

    6) Las mujeres con capacidad de procrear deberán obtener un resultado negativo en una prueba de embarazo en orina en el periodo basal y estar dispuestas a realizarse otras pruebas de embarazo adicionales durante el estudio. Las mujeres que se considera que no tienen capacidad de procrear incluyen las que no han alcanzado la menarquía, las mujeres posmenopáusicas (que son aquellas que no han tenido la menstruación durante 12 meses sin que exista una causa médica alternativa), o las que son estériles permanentemente debido a una histerectomía total, una salpingectomía bilateral o una ooforectomía bilateral.

    7) Las participantes con capacidad de procrear o los varones fértiles con parejas capaces de procrear que sean sexualmente activos deben acceder a utilizar un método anticonceptivo muy eficaz que determine el investigador, desde el período posterior a la firma del consentimiento informado hasta pasados 30 días después de la última dosis del fármaco del estudio.
    E.4Principal exclusion criteria
    1)Any known hypersensitivity to triheptanoin that, in the judgment of the investigator, places the subject at increased risk for adverse effects
    2)History of, or current suicidal ideation, behavior and/or attempts
    3)Pregnant and/or breastfeeding an infant
    4)Unwilling or unable to discontinue use of a prohibited medication (i.e. barbiturates, pancreatic lipase inhibitors) or other substance that may confound study objectives. Use of up to 3 concomitant AEDs is allowed, provided dose has been stable at least 14 days prior to Baseline.
    5)Use of any investigational product, drug or supplement (other than UX007) within 30 days prior to Baseline, or at any time during the study
    6)Has a condition of such severity and acuity, in the opinion of the investigator, that it warrants immediate surgical intervention or other treatment
    7)Has a concurrent disease or condition, or laboratory abnormality that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or introduce additional safety concerns (e.g. other concurrent neurological or psychiatric disorders)
    Hipersensibilidad conocida a la triheptanoína que, en opinión del investigador, aumente el riesgo del sujeto de sufrir efectos adversos.

    2) Antecedentes o presencia de ideación o conducta suicida y/o intentos de suicidio.


    3) Embarazo o lactancia

    4) Sujeto que no puede o no desea interrumpir el uso de un medicamento prohibido (esto es, barbitúricos, inhibidores de la lipasa pancreática) o de otras sustancias que podrían ocasionar una interpretación errónea de los objetivos del estudio. Se permite utilizar hasta tres FAE concomitantes, siempre que la dosis se haya mantenido estable durante al menos 14 días antes del periodo basal.

    5) Uso de algún producto, fármaco o complemento en investigación (que no sea UX007) durante los 30 días anteriores al periodo basal, o en cualquier momento durante el estudio.

    6) Presentar una enfermedad de tal gravedad y en una fase tan aguda que, en opinión del investigador, precisa una intervención quirúrgica de urgencia u otro tratamiento.

    7) Presentar una enfermedad o afección concurrente o una anomalía analítica que, en opinión del investigador, hace que el sujeto tenga un riesgo alto de incumplimiento terapéutico o de no completar el estudio, o interferiría en la participación en el estudio o introduciría problemas de seguridad adicionales (p. ej.: otras afecciones neurológicas o psiquiátricas concurrentes).
    E.5 End points
    E.5.1Primary end point(s)
    Frequency of adverse events (AE) and serious adverse events (SAE) assessed as related to study drug over the Treatment Extension Period
    La frecuencia de acontecimientos adversos (AA) y de acontecimientos adversos graves (AAG) que se consideren relacionados con el fármaco del estudio durante el período de extensión del tratamiento
    E.5.1.1Timepoint(s) of evaluation of this end point
    Month 0, 3, 6, 9, 12, 18, 24, 30, 36
    Meses 0, 3, 6, 9, 12, 18, 24, 30, 36
    E.5.2Secondary end point(s)
    •Frequency of seizures over the Treatment Extension Period
    •Measures of neurological function over the Treatment Extension Period
    •Health-related quality of life over the Treatment Extension Period
    La frecuencia de convulsiones durante el período de extensión del tratamiento
    Las mediciones de la función neurológica durante el período de extensión del tratamiento
    La calidad de vida relacionada con la salud durante el período de extensión del tratamiento
    E.5.2.1Timepoint(s) of evaluation of this end point
    •Frequency of seizures over the Treatment Extension Period :0, 3, 6, 9, 12, 18, 24, 30, 36
    •Measures of neurological function over the Treatment Extension Period :0, 3, 6, 9, 12, 18, 24, 30, 36
    •Health-related quality of life over the Treatment Extension Period :0, 3, 6, 9, 12, 18, 24, 30, 36
    La frecuencia de convulsiones durante el período de extensión del tratamiento: meses 0, 3, 6, 9, 12, 18, 24, 30, 36
    Las mediciones de la función neurológica durante el período de extensión del tratamiento: meses 0, 3, 6, 9, 12, 18, 24, 30, 36
    La calidad de vida relacionada con la salud durante el período de extensión del tratamiento: meses 0, 3, 6, 9, 12, 18, 24, 30, 36
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Denmark
    France
    Israel
    Italy
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS

    This open-label long-term safety and efficacy study will provide an opportunity for Glut1 DS patients to be treated with UX007 for up to 3 years (or until market approval) under a single standardized protocol for maintenance therapy and consistent safety monitoring.
    Última visita del último paciente

    Se trata de un estudio abierto para evaluar la seguridad y eficacia a largo plazo dando la oportunidad a los pacientes con síndrome por deficiencia del transportador de glucosa de tipo 1 de ser tratados con UX007 por un máximo de 3 años (o hasta que esté disponible comercialmente), todo ello en un protocol único estandarizado para terapia de mantenimiento y con una monitorización de seguridad consistente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 30
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Paediatric subjects
    pacientes pediátricos
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 40
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None

    This open-label long-term safety and efficacy study will provide an opportunity for Glut1 DS patients to be treated with UX007 for up to 3 years or until market approval, whichever occurs first.
    Ninguno
    Se trata de un estudio abierto para evaluar la seguridad y eficacia a largo plazo dando la oportunidad a los pacientes con síndrome por deficiencia del transportador de glucosa de tipo 1 de ser tratados con UX007 por un máximo de 3 años o hasta que esté disponible comercialmente (lo que ocurra primero)
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-05-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-05-11
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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